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Hormone hope for autism management

Two studies point to vasopressin as potential tool to improve social skills for people with autism spectrum disorder. Andrew Masterson reports.

To date, pharmacological interventions for the management of autism have met with limited success.

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Regulating a common hormone that influences kidney function has been shown to improve social functioning in adults and children with autism spectrum disorder (ASD).

Vasopressin, also known as antidiuretic hormone (ADH), is produced in the brain area known as the hypothalamus, and functions to increase reabsorption in the circulatory system of water cleaned by the kidneys. It also plays a role in raising arterial blood pressure.

Now, in two trials, researchers have also linked it to social behaviours in people with autism.

Scientists led by Federico Bolognani from the Roche Innovation Centre in Basel, Switzerland, conducted a phase II clinical trial involving 223 adult men with moderate to severe ASD, testing the effects of a vasopressin inhibitor drug called balovaptan.

In a second experiment, a team led by Karen Parker from Stanford University in the US administered vasopressin (or a placebo) directly to 30 autistic children aged between six and 12, for a period of four weeks.

Both studies are published in the journal Science Translational Medicine.

Bolognani’s cohort received one of three different doses of balovaptan, or a placebo. The participants receiving the two highest doses of the drug showed improvements in socialisation, adaptive behaviour and daily living skills, compared with those on a low dose of the drug or the placebo.

Parker’s team relied on parent observations for their results. On that basis, the researchers report that the 17 children receiving vasopressin also showed enhanced social behaviours compared to the 13 who received the placebo.

Both sets of researchers also noted that the treatments were well tolerated, produced no side-effects, and also reduced other symptoms associated with ASD, such as anxiety.

The findings are potentially significant, because to date other pharmacological interventions for the management of ASD have been largely unsuccessful.

“Further studies are needed to evaluate the potential for balovaptan to improve social communication and social interaction deficits within broader ASD patient populations, including children,” concludes Bolognani’s team.